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Title of At Risk for Huntington's Disease: "At Risk for Huntington's Disease"
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As researchers have hypothesized that lowering (reducing) the amount of abnormal huntingtin protein in the brains of people afflicted with Huntington’s disease hits at the root causes of the disease, leading companies have sought to develop drugs in response, like ...
Delaying but not blocking AMT-130, uniQure’s emerging gene therapy to slow Huntington’s disease progression, the U.S. Food and Drug Administration (FDA) “strongly recommended” that the company conduct a full-blown, Phase 3 clinical trial, rather than asses...
AMT-130, the uniQure gene therapy that successfully slowed the progression of Huntington’s disease before being
With as many as 15 million people estimated to lose their medical coverage becaus...
On January 22 Huntington’s disease advocacy organizations delivered two petitions to the U.S. Food and Drug Administration (FDA), demanding that the agency reverse its rollback on uniQure’s application for its HD gene therapy.
In Sep...